Many pundits worried that last week’s rapid purge of information about diversity and inclusion in clinical trials for cancer drugs on the FDA website signaled that the FDA no longer requires diversity in clinical trial participants. It may not be that simple.
The need for diversity of clinical trial participants is not a political issue, but a scientific necessity. Different racial and ethnic groups may respond differently to the same medical treatment due to genetic variations. For example, certain populations metabolize drugs at different rates, affecting their efficacy and safety. Many diseases disproportionately affect specific demographic groups. For example, African Americans have a higher prevalence of hypertension, and chronic kidney disease, and are more likely to suffer from complications of cardiovascular disease. Likewise, Hispanic and Native American populations have higher rates of Type 2 diabetes. Lifestyle, diet, and environmental exposures can significantly influence treatment outcomes. Social determinants of health, such as access to healthcare, economic status, and geographical location, can impact drug effectiveness and patient adherence to treatment. Including diverse participants helps researchers understand the broader implications of these factors in real-world settings, and for the prescribers to tailor treatment approaches to meet their unique medical needs once the drug is approved.
Over the last decade, the FDA released several guidance documents and initiatives to encourage and support sponsors in their efforts to recruit diverse populations in their clinical trials. These include guidance on proactive strategies for increasing trial diversity, such as community outreach, flexible trial designs, and removing barriers like transportation and language limitations. Also, under the Food and Drug Omnibus Reform Act (FDORA) of 2022, sponsors of clinical trials are required to submit a diversity action plan outlining their strategy to enroll participants from underrepresented racial, ethnic, and gender groups. The FDA also releases Drug Trial Snapshots that provide the demographic breakdown of participants in clinical trials for newly approved drugs to educate prescribers and patients about the trials that formed the basis of approval.
The FDA’s expectation for the inclusion of diverse populations in clinical trials is based on the need for trials to reflect the demographic diversity of the U.S. population. However, the FDA never rejected a new drug for lack of diversity in the participants in clinical trials whose data was used to support approval. Rather most drugs are still approved based on data from trials with non-diverse populations.
Although the FDA’s senior management is political, almost all the rest of its 18,000 employees are career regulators, whose job descriptions have not changed. They are still required to make decisions based on scientific rationale and credible data. While we might see some policy changes at the higher levels of the FDA mostly around leadership initiatives, the common sense logic behind the need for diversity in clinical trials is still relevant. From a strategic perspective, do not make changes to your ongoing recruitment efforts, still develop the diversity action plan, and be prepared to defend how you are ensuring diversity in the participants in your clinical trial.
Diversity in clinical trials is not merely a regulatory issue; it is a fundamental necessity for the advancement of precision medicine and health equity. Without adequate representation, treatments may fail to meet the needs of all patients, exacerbating existing healthcare disparities. And healthcare disparities affect the entire population, not just the ones being disparaged.
